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STAT1-related autoimmune enteropathy and endocrinopathy-susceptibility to chronic infections syndrome

ORPHA:391487· ICD-10 K63.9

Definition

An extremely rare, autosomal dominant immunological disorder characterized by variable enteropathy, endocrine disorders (e.g. type 1 diabetes mellitus, hypothyroidism), immune dysregulation with pulmonary and blood-borne bacterial infections, and fungal infections (chronic mucocutaneous candidiasis) developing in infancy. Other manifestations include short stature, eczema, hepatosplenomegaly, delayed puberty, and osteoporosis/osteopenia.

Prevalence
<1 / 1 000 000
Inheritance
Autosomal dominant
Age of onset
Infancy